Rare disease (orphan) designations

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This page allows you to find information on rare disease (orphan) designations based on applications that have been assessed by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP).

An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

For more information, see Orphan designation.

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EPAR Search results
Active substance Disease / condition Date of decision Decision Medicine name

Herpes simplex 1 virus-thymidine kinase and truncated low affinity nerve growth factor receptor transfected donor lymphocytes

Adjunctive treatment in haematopoietic cell transplantation 2003-10-20 Positive Zalmoxis

Carboxypeptidase G2

Adjunctive treatment in patients at risk of methotrexate toxicity 2003-02-03 Positive  

Plerixafor

Adjunctive treatment to cytotoxic therapy in acute myeloid leukaemia 2011-12-09 Withdrawn  

Somatropin

AIDS wasting 2000-08-08 Positive  

Complement factor H

Atypical haemolytic uraemic syndrome 2007-01-26 Withdrawn  

Estradiol hemihydrate and progesterone

Bronchopulmonary dysplasia in premature neonates of less than 30 weeks of gestational age 2005-04-11 Positive  

Thiotepa

Conditioning treatment prior to haematopoietic progenitor cell transplantation 2007-01-29 Positive Tepadina

Treosulfan

Conditioning treatment prior to haematopoietic progenitor cell transplantation 2004-02-22 Positive  

Busulfan

Conditioning treatment prior to haematopoietic-progenitor-cell transplantation 2000-12-29 Expired Busilvex

Ex-vivo-expanded autologous human corneal epithelium-containing stem cells

Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns 2008-11-06 Positive Holoclar

68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid (satoreotide trixozetan)

 

Diagnosis of gastro-entero-pancreatic neuroendocrine tumours 2014-02-19 Positive  

Gallium (68Ga)-edotreotide

Diagnosis of gastro-entero-pancreatic neuroendocrine tumours 2015-03-19 Positive SomaKit TOC

Gallium (68Ga)-pasireotide tetraxetan

Diagnosis of gastro-entero-pancreatic neuroendocrine tumours 2011-10-27 Positive  

Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide

Diagnosis of gastro-entero-pancreatic neuroendocrine tumours 2014-02-19 Positive  

68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2

Diagnosis of gastrointestinal stromal tumours 2016-12-12 Positive  

4-[123I] Iodo-L-phenylalanine

Diagnosis of glioma 2006-07-25 Positive  

Florilglutamic acid (18F)

Diagnosis of glioma 2016-03-21 Positive  

Fluciclovine (18F)

Diagnosis of glioma 2015-04-24 Positive  

Tetrofosmin

Diagnosis of glioma 2016-09-20 Positive  

Florilglutamic acid (18F)

Diagnosis of hepatocellular carcinoma 2016-03-21 Positive  

Metastable technetium 99 [99mTc] demogastrin 2

Diagnosis of medullary thyroid carcinoma 2006-08-28 Positive  

Folic acid to be used with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine

Diagnosis of positive folate-receptor status in ovarian cancer 2012-09-10 Withdrawn Neocepri

N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine and folic acid

Diagnosis of positive folate-receptor status in ovarian cancer 2012-02-09 Withdrawn  

N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine to be used with folic acid

Diagnosis of positive folate-receptor status in ovarian cancer 2012-09-10 Positive Folcepri

Iodine (123I) Serum Amyloid P

Diagnosis of the extent of histologically proven amyloidosis 2003-02-14 Positive